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Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.


 
Posted on: 15 November 2014, source: Nature
Tiny changes in DNA can have huge consequences. For years, scientists have been trying to 'fix' these mutations in the hope of treating and potentially curing some of humanity's most devastating genetic diseases. After some tragic early setbacks (see Nature 420, 116–118; 2002), techniques that allow precise genetic manipulation have created a surge of research.
Although most existing treatments for genetic diseases typically only target symptoms, genetic manipulation or 'gene therapy' goes after the cause itself. The approach involves either inserting a functional gene into DNA or editing a faulty one that is already there, so the conditions most likely to prove curable are those caused by a single mutation. Sickle-cell disease is a perfect candidate: it is caused by a change in just one amino acid at a specific site in the β-globin gene. This results in the production of abnormal haemoglobin proteins that cause the red blood cells that house them to twist and become sickle shaped. The distorted cells get sticky, adhere to each other and block blood vessels, preventing oxygenated blood from flowing through

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Posted on: 6 November 2014, source: ieet.org
Maria Konovalenko and team put together a list of popular science video lectures on gene therapy – one of the most promising molecular medicine directions. What makes this approach different is that nucleic acid molecules, DNA and RNA, are used as therapeutic agents.

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Posted on: 9 October 2014, source: healio.com
A majority of boys with X-linked severe combined immunodeficiency experienced T-cell recovery and infection clearance after undergoing gene therapy with a self-inactivating gamma-retrovirus vector, according to study results. Salima Hacein-Bey-Abina, PharmD, PhD, of the department of biotherapy at Hôpital Necker – Enfants Malades in Paris, and colleagues sought to modify a Moloney murine leukemia virus-based gamma-retrovirus vector that expressed interleukin-2 receptor gamma-chain complementary DNA

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Posted on: 28 August 2014, source: Chemistry World
Dutch scientists have built a simple model of viruses’ protective coats in an attempt to create viral mimics that could fight diseases, as opposed to causing them. Rather than copying natural proteins, Renko de Vries from Wageningen University and his team designed and built a three-part protein from scratch that self-assembles around DNA.
‘The protein is exceedingly simple in its primary and secondary structure, yet captures the essence of self-assembly for the tobacco mosaic virus,’ de Vries tells Chemistry World. This knowledge could enable superior vehicles for getting DNA and RNA into cells, for example for gene therapy, and templates for improved DNA machines. ‘You could probably do the same with supramolecular chemistry,’ de Vries adds, ‘but the protein approach has the beauty that you can expand in the direction of synthetic biology.’

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Posted on: 21 August 2014, source: Sci-News.com
Dr Juliana Small of the University of Pennsylvania, Drs Raj Kurupati, Xianqyang Zhou and their colleagues from the Wistar Institute have developed a novel adenoviral vector for delivery of multiple transgenes.

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Posted on: 6 August 2014, source: The Independent
Scientists have performed a “seamless” correction to a faulty gene behind an inherited form of anaemia using a revolutionary new technique in genome editing that could transform the treatment of many genetic diseases. Two mutations in the haemoglobin gene of a patient with beta thalassemia – which can cause severe anaemia – were corrected without any errors using the Crispr technique of genome editing, the researchers said. The experiment involved converting the patient’s skin cells into stem cells in the laboratory so that the faulty gene could be corrected before the stem cells were allowed to mature into red blood cells. Without the genome correction, the red cells would have become deformed and sickle-shaped as a result of the defective haemoglobin gene.

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The market approval of gene therapy product Glybera in Europe will accelerate regulatory approvals of other gene medicines
 
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